Regeneron licenses gene-editing technology in nine-figure deal

Regeneron Pharmaceuticals Inc. will make a $75 million initial payment and provide $50 million in equity financing to a Cambridge, Mass., company as part of a 6-year licensing and collaboration agreement to develop in vivo drugs using a gene-editing technology that has been called the biggest biotech discovery of the century.

Intellia Therapeutics, Inc. gave Westchester-based Regeneron, New York”™s largest biotech employer, the exclusive right to discover and develop treatments using Intellia”™s 4-year-old CRISPR/CAS technology platform against up to 10 targets. The research will focus primarily on therapies for diseases that can be treated by editing genes in the liver.

The companies in a press release said Intellia will receive a $75 million upfront payment and could collect significant milestone and royalty payments on potential Regeneron products. The companies have agreed to co-develop and co-commercialize a certain number of targets generated during the collaboration. Transthyretin amyloidosis, a neurodegenerative disease, is the first target to be jointly developed and potentially commercialized by the companies.

Discovered by an Intellia founder in 2012, the CRISPR/Cas9 technology can cut DNA in precise locations so as to selectively knock out, repair or insert specific genetic sequences. Regeneron and Intellia scientists said it potentially can be applied across multiple therapeutic areas including autoimmune diseases, metabolic and blood disorders, cancer and rare and genetic-based diseases.

Dr. George D. Yancopoulos, Regeneron”™s chief scientific officer of Regeneron and president of Regeneron Laboratories, in the recent announcement noted that researchers in the Regeneron Genetics Center, at the company”™s headquarters on the Landmark at Eastview campus, has already been identifying important human genetic targets in treating diseases. “We believe combining these capabilities with Intellia’s technology holds real promise for serious diseases that have been historically difficult to address, and expands our ability to help patients where antibody-based therapies may not be the optimal solution,” he said.

According to Intellia”™s website, geneticists have used the gene-slicing technology to rid mice of muscular dystrophy and cure them of a rare liver disease, make human cells immune to HIV, correct sickle cell anemia and alter cancer cells to make them more susceptible to chemotherapy. The technological platform is said to be ideal for “developing an entirely new class of genetic medicines” and led the MIT Technology Review to describe it as “the biggest biotech discovery of the century.”

Nessan Bermingham, founder and CEO of Intellia Therapeutics said of the licensing and collaboration agreement, “Regeneron’s focus on advancing science to medicine is an excellent fit with Intellia’s approach, and together, we aim to bring potential cures to patients who are suffering from life-threatening rare diseases and genetic diseases.”